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Objectives: The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making. Materials and Methods: A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries. A descriptive and statistical analysis was performed to identify correlations between country characteristic, HTA procedures, drug indication and positive recommendations or reimbursement decisions for ODs. Results: Safety assessment for ODs was mandatory in 10 countries, while it was optional in one country (Italy) and not required in one country (Iceland). Efficacy assessment for ODs was mandatory in 11 countries and not required in one country (Iceland). The impact of safety and efficacy assessment on reimbursement decisions was rated as high in 10 countries and as low in one country (Germany). Dedicated OD legislation and policies were reported in seven countries. In two countries (Belgium, Iceland), the HTA was not mandatory, and in one country (Germany), it only had an informative function. A positive recommendation (from an HTA agency or advisory body) guaranteed reimbursement in four countries, while a negative recommendation excluded reimbursement only in one country (Iceland). The proportion of reimbursed ODs ranged from 23.5% in Iceland to 86% in Germany (p < 0.001). ODs with ATC code L represented the largest group of medicines (n = 49). They were also very frequently reimbursed ODs in the countries studied, with a mean of 61.8% (p < 0.001). Conclusion: European countries differ in terms of the impact of clinical issues and additional clinical aspects on the reimbursement policy for ODs. Reimbursement decisions were affected by OD-specific legislation, policies, and EMA authorization status. HTA dossiers and procedures significantly influenced reimbursement decisions, although some ODs were reimbursed regardless of the positive or negative recommendations. ATC codes were significantly correlated with reimbursement status and positive recommendation. [ABSTRACT FROM AUTHOR]

Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe. This paper elicited the opinions of European stakeholders on how HTA can be improved to facilitate access. Methods: A scoping review identified HTA features that influence access to medicines within markets and areas for improvement, while three access dimensions were identified (availability, affordability, timeliness). Using the Delphi method, we elicited the opinions of European stakeholders to validate the literature findings. Results: Nineteen participants from 14 countries participated in the Delphi panel. Thirteen HTA features that could be improved to optimise access to medicines in Europe were identified. Of these, 11 recorded a positive impact on at least one of the three access dimensions. HTA features had mostly a positive impact on timeliness and a less clear impact on affordability. 'Early scientific advice' and 'clarity in evidentiary requirements' showed a positive impact on all access dimensions. 'Established ways to deal with uncertainty during HTA' could improve medicines' availability and timeliness, while more 'reliance on real-world evidence' could expedite time to market access. Conclusions: Our results reiterate that increased transparency during HTA and the decision-making processes is essential; the use of and reliance on new evidence generation such as real-world evidence can optimise the availability of medicines; and better collaborations between regulatory institutions within and between countries are paramount for better access to medicines. [ABSTRACT FROM AUTHOR]

There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology. • Health technology assessments of paediatric oncology drugs vary greatly within Europe. • The timing of HTA evaluation adds to the delay of patient access. • Differences between adult and paediatric trial methodology should be considered. • Multistakeholder collaboration between EMA, academia, pharmaceutical companies, patients advocates and HTAs is needed. [ABSTRACT FROM AUTHOR]

Résumé: Le dernier congrès de l'ISPOR Europe (International society for pharmacoeconomics and outcomes research) s'est tenu à Copenhague du 2 au 6 novembre 2019. Un groupe de pharmaciens, médecins et économistes en santé en synergie avec un groupe d'étudiants en master 2 de médico-économie présents à Copenhague, ont choisi de vous en restituer les messages forts. Cette synergie de groupe vise à développer « l'approche de la médico-économie sous toutes ses facettes ». Dans cet article, les auteurs évoquent les questions issues des différentes sessions et/ou posters, notamment l'évaluation des produits de santé : comment accélérer l'accès au marché des traitements tout en garantissant la pertinence clinique et la maîtrise des dépenses ? The last congress of ISPOR Europe (International society for pharmacoeconomics and outcomes research) was held in Copenhagen from 2 to 6 November 2019. A group of pharmacists, physicians and health economists in synergy with a group of students in Master 2 of medico-economics attending the conference have chosen to share some of the highlights. This group synergy aims to develop "the medico-economic approach in all its facets". In this paper, the authors discussed the following questions from the different sessions and/or posters, in particular the evaluation of health products: how to accelerate access to the treatment market while ensuring clinical relevance and control of spending? [ABSTRACT FROM AUTHOR]

Access to medicines treating rare diseases ('orphan medicines') has proven challenging due to high prices and clinical uncertainty. To optimise market access to these medicines, some healthcare systems are implementing specialised pathways and/or processes during marketing authorisation (MA) and/or health technology assessment (HTA). Comparing one setting where these medicines are classed as "orphan" (Scotland) to another where they considered "non-orphan" (Canada), this study aims to explore whether the presence of specialised pathways and processes at MA and HTA levels is associated with more favourable funding recommendations and faster time to market access. A matched sample of 116 medicine-indication pairs with MA approval from 2001 to 2019 in Europe and Canada was identified, and publicly available sources were used for data extraction. Descriptive statistics were used for data analysis. All medicines were commercially marketed in both countries, except one instance in Scotland. In Scotland, more orphan medicines (68.1%) had a favourable HTA recommendation than in Canada (60.4%), while Canada issued more negative HTA recommendations (20.7%) than Scotland (15.5%). Low levels of agreement on HTA recommendations and the main reasons driving recommendations were found between settings. In both countries, medicines with specialised MA approval were less likely to receive negative HTA recommendations than medicines with standard MA. Time to market access was faster in Canada than Scotland, though medicines with specialised MA approval had slower timelines than medicines with standard MA approval in both countries. However, it is unclear whether the presence of orphan designation and HTA specialised processes alone could result in favourable funding recommendations without accounting for other healthcare system-related factors and differences in the decision-making processes across settings. Holistic approaches and better alignment of evidentiary requirements across regulators are needed to optimise access to orphan medicines. • Canada approved more orphan medicines through specialised pathways than Scotland. • Less negative HTA outcomes for medicines with specialised MA in both countries. • Less negative HTA recommendations for orphan medicines in Scotland than Canada. • Low levels of agreement in HTA for orphan medicines between Scotland and Canada. • Time to market access occurred considerably faster in Canada than Scotland. [ABSTRACT FROM AUTHOR]

Background: Several Central and Eastern European (CEE) countries require cost-utility analyses (CUAs) to support reimbursement formulary listing. However, CUAs informed by local evidence are often unavailable, and the cost-effectiveness of the several currently reimbursed biologicals is unclear. Aim: To estimate the cost-effectiveness as multiples of per capita GDP/quality adjusted life years (QALY) of four biologicals (infliximab, etanercept, adalimumab, golimumab) currently reimbursed in six CEE countries in six inflammatory rheumatoid and bowel disease conditions. Methods: Systematic literature review of published cost-utility analyses in the selected conditions, using the United Kingdom (UK) as reference country and with study selection criteria set to optimize the transfer of results to the CEEs. Prices in each CEE country were pro-rated against UK prices using purchasing power parity (PPP)-adjusted per capita GDP, and local GDP per capita/QALY ratios estimated. Results: Central and Eastern European countries list prices were 144-333 % higher than pro rata prices. Out of 85 CUAs identified by previous systematic literature reviews, 15 were selected as a convenience sample for estimating the cost-effectiveness of biologicals in the CEE countries in terms of per capita GDP/QALY. Per capita GDP/QALY values varied from 0.42 to 6.4 across countries and conditions (Bulgaria: 0.97-6.38; Czech Republic: 0.42-2.76; Hungary: 0.54-3.54; Poland: 0.59-3.90; Romania: 0.77-5.07; Slovakia: 0.55-3.61). Conclusion: While results must be interpreted with caution, calculating pro rata (cost-effective) prices and per capita GDP/QALY ratios based on CUAs can aid reimbursement decision-making in the absence of analyses using local data. [ABSTRACT FROM AUTHOR]

In 2020, European Network for Health Technology Assessment (EUnetHTA) published a relative effectiveness analysis (REA) of Pretomanid in combination with Bedaquiline and Linezolid for the treatment of extensively drug-resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB) (REA PTJA14). This REA may have a significant value for low- and middle-income countries (LMICs) outside Europe, particularly those with a high burden of drug-resistant TB. This commentary focuses on whether the REA PTJA14 can be transferred and to what extent a REA can be translated to LMICs context outside Europe. We found that the assessments on the clinical effectiveness and risks of bias reported in REA PTJA14 are useful for LMICs outside Europe. The highly standardized management of TB will support the applicability of the REA to LMICs outside of Europe. Transferring this REA can reduce workload and efficiently use limited resources to conduct health technology assessment (HTA). However, the transfer should consider several critical issues, including variations in health system delivery and clinical practice and setting-specific constraints. In the TB context, the differences in the current standard treatment for XDR or nonresponsive MDR TB, resources availability for drug-resistant TB management, and how healthcare is delivered in the countries can complicate the applicability of the REA PTJA14. Given that LMICs have limitations in doing HTA, it is now critical to develop standard guidelines for transferring REA or other HTA results from high-income countries or other LMICs to maximize the benefits of the REA for LMICs outside Europe.

Patient involvement in the process of producing health technology assessments has become increasingly important. In the European Network for Health Technology Assessment (EUnetHTA), several approaches to patient involvement were explored. The outcome was a document on "Patient Input in Relative Effectiveness Assessments" that is available for access and was published in 2019.The aim of this article is to analyze the experience gained by EUnetHTA in patient involvement for EUnetHTA assessment production, describe and quantify the approaches used, and outline the challenges and avenues for the improvement of current processes.Patients were involved in twenty-three of thirty-six pharmaceutical and other technology EUnetHTA assessments from June 2016 until the end of November 2019. Approaches to patient involvement included using a patient input template, one-on-one conversations, group conversations, scoping meeting with patients, and other approaches.Although it is recognized that patient involvement is important to understand the needs of the target patient population, challenges remain with timely patient involvement. Additionally, further efforts are needed to guide assessment teams on how to implement and enhance the visibility of patient input in assessments.

Introduction: The new European Union (EU) Regulations on medical devices and on in vitro diagnostics provide manufacturers and Notified Bodies with new tools to improve pre-market and post-market clinical evidence generation especially for high-risk products but fail to indicate what type of clinical evidence is appropriate at each stage of the whole lifecycle of medical devices. In this paper we address: i) the appropriate level and timing of clinical evidence throughout the lifecycle of high-risk implantable medical devices; and ii) how the clinical evidence generation ecosystem could be adapted to optimize patient access., Areas Covered: The European regulatory and health technology assessment (HTA) contexts are reviewed, in relation to the lifecycle of high-risk medical devices and clinical evidence generation recommended by international network or endorsed by regulatory and HTA agencies in different jurisdictions., Expert Opinion: Four stages are relevant for clinical evidence generation: i) pre-clinical, pre-market; ii) clinical, pre-market; iii) diffusion, post-market; and iv) obsolescence & replacement, post-market. Each stage has its own evaluation needs and specific studies are recommended to generate the appropriate evidence. Effective lifecycle planning requires anticipation of what evidence will be needed at each stage.

The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with stakeholders to receive feedback on its activities, processes, and outputs produced. The fourth edition of the EUnetHTA Forum brought together representatives of HTA bodies, patient organizations, healthcare professionals (HCPs), academia, payers, regulators, and industry. The aim of this paper is to provide an overview of the highlights presented at the 2019 EUnetHTA Forum, reporting the main items and themes discussed in the plenary panel and breakout sessions. The leading topic was the concept of unmet medical need seen from different stakeholders' perspectives. Breakout sessions covered the joint production of assessment reports and engagement with payers, patients, and HCPs. Synergies, pragmatism, and inclusiveness across Member States and stakeholders were emphasized as leading factors to put in place a collaboration that serves the interest of patients and public health in a truly European spirit.

Introduction: Public health (PH) interventions are crucial for ensuring sustainable healthcare services. Nevertheless, they represent a neglected area in the field of health technology assessment (HTA) due to various methodological issues and their complex design that goes beyond clinical setting. The present study provides an environmental scan of HTA initiatives related to the assessment of PH technologies on a global level. Methods: We conducted a cross-sectional survey among 85 HTA-related European and international societies, health bodies, and networks from September 2018 to January 2019. The questionnaire contained four sections and 18 questions regarding activities related to the evaluation of PH technologies, information on existing PH technologies, and methodologies of assessment as well as barriers and facilitators to reaching a decision and implementing a PH technology. Results: Among 52 survey responses, the majority of the respondents came from European countries (35%), followed by North American (27%), and South American (19%) countries. The main type of organizations covered by our survey included HTA agencies, public administrations, and research institutes. Seventy-one % of the institutions reported engagement in any aspect of HTA in the area of PH ( N = 37). Among those, 81% evaluated less than 5 PH technologies from 2013 to 2018. The most common barriers for reaching a decision on PH technologies were lack of data, conflicting stakeholder priorities, and methodological issues. A total of 76 PH interventions were reported, and most cited initiatives were related to chronic disease screening, prevention of infectious diseases, and maternal, prenatal, and neonatal screening. Conclusion: Our survey reported a rather limited involvement of HTA in the evaluation of PH technologies. In particular, an evaluation of behavioral and lifestyle interventions remains extremely rare. The implementation of collaborative HTA approaches in the setting of PH practice and policy needs to be prioritized and further strengthened. Moreover, ensuring reliable data structures and consolidation of HTA methods for the evaluation of PH technologies will be crucial for tackling the enormous burden of non-communicable diseases in societies., (Copyright © 2020 Stojanovic, Wübbeler, Geis, Reviriego, Gutiérrez-Ibarluzea and Lenoir-Wijnkoop.)

Objectives: The primary objective of this study was to compare the availability and access of orphan medicinal products (OMPs) in the devolved nations in the United Kingdom (UK), France, Germany, Italy and Spain. Availability is defined as the possibility to prescribe OMPs. Access refers to their full or partial reimbursement by the public health service., Methods: Data were collated on: marketing authorisations, Health Technology Assessment (HTA) decisions, commissioning, and reimbursement decisions, and respective dates of these events for all the OMPs centrally authorised. Indicators of availability of and access to OMPs were calculated in each country and compared., Results: We found that since the implementation of the OMPs Regulation in 2000 to end of May 2016, 143 OMPs obtained a marketing authorisation in the European Union. These OMPs are most widely accessible in Germany and France. In the other countries between 30 and 60% of OMPs are reimbursed. In particular in England, less than 50% of centrally authorised OMPs are routinely funded by the NHS, with one-third of these recommended by NICE. In Germany reimbursement is automatically granted to all medicines which receive a marketing authorisation, immediately after authorisation - but since 2011, there is an evaluation and potentially a pricing negotiation between companies and sickness funds (third party payers). In the other countries, the shortest time from authorisation to a reimbursement decision is observed in Italy and France where it takes 18.6 and 19.5 months respectively on average., Conclusions: Marketing authorisation granted to OMPs is only the first step, as medicines reach patients when reimbursement decisions are implemented by national health systems (this applies to non-OMPs too). We found that more than a half of centrally authorised OMPs were available in the five selected countries, but that access to patients was further restricted by different national reimbursement policies, especially in the UK, Italy and Spain.

The article presents information on health technology assessment (HTA) organizations in various countries. It is stated that health technology assessment regarding health policy decisions has received a more favorable response in Europe than in the U.S.; and HTA has become a political priority in Europe. It is stated that compared to Europe, the U.S. has a more privately oriented and decentralized health-care system.